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X4 Pharmaceuticals Inc is clinical-stage biopharmaceutical company. It is focused on restoring the immune system function by developing therapeutics for the treatment of rare diseases. Its molecule therapeutics are antagonists of chemokine receptor CXCR4, which have the potential to treat a broad range of rare diseases, including primary immunodeficiencies (PIs) and cancer. Its product candidate, X4P-001, has completed a Phase 2 trial in patients with a rare genetic PI called Warts. The company is also developing X4P-002, a CXCR4 antagonist that has properties that could enable penetration of the blood-brain barrier and provide appropriate therapeutic exposures to treat brain cancers, including glioblastoma multiforme.